Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses the implications of a matched targeted therapy approach in pediatric leukemia.

At the 2018 ASH Annual Meeting, preliminary data were presented on a study in which investigators sequenced samples of pediatric patients’ leukemia in an attempt to identify targetable mutations. If a target was found, investigators tried to pair it with a corresponding therapy. Pending further results, this may inform the integration of novel therapies into clinical practice for patients with pediatric leukemia, explains Pikman. This may fill a large unmet need, as new drugs are tested in adults before they are ever brought to pediatric patients, she adds.

Based on the initial results of the study, pediatric leukemia behaves differently than adult leukemia, says Pikman. As such, although novel drugs that are proven to be ineffective in adult patients are halted for further development, they may be effective in pediatric patients. Putting a genomic characterization of pediatric leukemia in place will help inform which drugs are needed as well as shape how these drugs are tested in trials for pediatric patients.

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Dr. Pikman on Implications of a Matched Targeted Therapy Approach in Pediatric Leukemia

Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses a trial attempting to match pediatric patients with more precise therapy for acute leukemia. Early findings were reported at the 2018 ASH Annual Meeting.

At the time of enrollment, pediatric patients had a sample of their leukemia sequenced and those data were reviewed by a panel of pediatric oncologists, therapeutic experts, and pathologists. Pikman says this process was to determine if researchers were able to identify any targetable mutations. If a targetable recommendation was made, the information was passed along to try to match a corresponding therapy.

This was a feasibility study, and Pikman says the next steps would include therapeutic studies testing the efficacy of the targeted agents. Researchers are discovering a number of novel genomic findings and identifying their significance to see whether therapy can be tailored towards them.

Yana Pikman, MD, a physician in the Cancer and Blood Disorders Center at Dana-Farber Cancer Institute and instructor of pediatrics at Harvard Medical School, discusses a trial attempting to match pediatric patients with more precise therapy for acute leukemia.

Yana Pikman, MD

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Dr. Pikman on Implications of a Matched Targeted Therapy Approach in Pediatric Leukemia

Dr. Pikman on Matching Pediatric Patients With More Precise Leukemia Therapy